Neurofibromatosis type 1

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At a Glance

Live from database

2FDA treatments

30Recruiting trials

10Travel grants

Regulatory Watchboard

10 events

Jul 2026Study to Determine Optimal Dose, Evaluate the Efficacy and Safety of PRG-N-01 in Patients With Neurofibromatosis Type II

PRG Science & Technology Co., Ltd. — PHASE1, PHASE2

TrialNOT YET RECRUITING

Feb 2026A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)

National Cancer Institute (NCI) — NA

TrialRECRUITING

Feb 2026Assessment of Patient Experience With Auto-Captioning Glasses in NF2-Related-Schwannomatosis

Massachusetts General Hospital — NA

TrialRECRUITING

Feb 2026Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1

Johns Hopkins University — PHASE1

TrialRECRUITING

Jan 2026Phase 1/2 Study of Mirdametinib + Vinblastine for Newly Diagnosed/Previously Untreated PLGG + Activation of MAPK

St. Justine's Hospital — PHASE1, PHASE2

TrialRECRUITING

Nov 2025Prevalence, Clinical Characteristics, Progression, and Management of Neurofibromatosis Type 1 in Egypt (NF1-Egy)

AstraZeneca — NA

TrialRECRUITING

Jun 2025Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1

Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. — PHASE1, PHASE2

TrialNOT YET RECRUITING

Jun 2025Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsia

AstraZeneca — NA

TrialRECRUITING

May 2025PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas

Pasithea Therapeutics Corp. — PHASE1

TrialRECRUITING

Apr 2025Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study

National Cancer Institute (NCI) — NA

TrialRECRUITING

Data from FDA regulatory filings and ClinicalTrials.gov. Updated periodically.

Financial Assistance

12 programs

FINANCIAL LANDSCAPE SUMMARY

12

Total programs

9

Open now

$5K

Available/year

1

Copay cards

10

Travel grants

Copay Assistance1

KOSELUGO

AstraZeneca

OpenContact for detailsApply ↗

Patient Assistance Programs1

Gomekli

SpringWorks Therapeutics (EMD Serono / Merck KGaA)

OpenContact for detailsApply ↗

Travel Grants10

Assistance Fund: Neurofibromatosis

OpenContact for detailsApply ↗

Neurofibromatosis medications copay assistance - PAN Foundation

PAN Foundation

OpenContact for detailsApply ↗

PAN Foundation Neurofibromatosis Disease Fund

PAN Foundation

OpenContact for detailsApply ↗

PAN Foundation Neurofibromatosis Fund

PAN Foundation

ClosedContact for detailsApply ↗

PAN Foundation Neurofibromatosis Grant

PAN Foundation

Contact for detailsApply ↗

PAN Foundation Neurofibromatosis Patient Assistance Grant

PAN Foundation

ClosedContact for detailsApply ↗

PAN Foundation Neurofibromatosis Premium Assistance Fund

PAN Foundation

OpenContact for detailsApply ↗

PAN: Neurofibromatosis (Premium Assistance)

OpenUp to $5K/yrApply ↗

The Assistance Fund (TAF)

The Assistance Fund

OpenContact for detailsApply ↗

The Assistance Fund (TAF) Neurofibromatosis Financial Assistance Program

OpenContact for detailsApply ↗

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Approved Treatments

2 FDA-approved

Gomekli

(mirdametinib)Orphan drug

SpringWorks Therapeutics (EMD Serono / Merck KGaA)

Kinase Inhibitor [EPC]

12.1 Mechanism of Action Mirdametinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2). MEK1/2 proteins are upstream regul...

Approved Feb 2025FDA label ↗

Koselugo

(selumetinib)Orphan drug

AstraZeneca / Alexion

Kinase Inhibitor [EPC]

12.1 Mechanism of Action Selumetinib is an inhibitor of mitogen-activated protein kinase kinases 1 and 2 (MEK1/2). MEK1/2 proteins are upstream regula...

Approved Apr 2020FDA label ↗

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Clinical Trial Landscape

30 active trials

2Phase 3

5Phase 2

9Phase 1

12N/A

2Unknown

30Total recruiting

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Recent News & Research

1 article

Mult Scler J Exp Transl ClinMar 1, 2026

The interconnection of multiple sclerosis and neurofibromatosis type 1: A single institution retrospective study and systematic review

Published in Mult Scler J Exp Transl Clin. Bazer DA et al.

Browse all Neurofibromatosis type 1 news →

Specialist Network

Top 6 by expertise

Zhichao Wang

Xuanwu Hospital, Capital Medical University

📍 Beijing, Beijing Municipality

Score 31908 pubs

Wenbin Li, MD

Cancer Hospital Chinese Academy of Medical Sciences

📍 Beijing, Beijing Municipality

Carlos G Romo, MD

Johns Hopkins University

📍 Baltimore, Maryland

Shivani Ahlawat, PhD

Johns Hopkins University

📍 Baltimore, Maryland

Mary Kay Koenig, MD

The University of Texas Medical School at Houston

📍 Houston, Texas

Score 224 trials

Miriam Bornhorst, MD

Children's National Health System

📍 Washington D.C., District of Columbia

Score 222 trials

View all Neurofibromatosis type 1 specialists →

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